New FDA Basics Video Available about Orphan Drugs and the Fight Against Rare Diseases

As part of FDA Basics, FDA has posted a new video with Dr. Tim Cote, director of FDA’s Office of Orphan Products Development. Dr. Cote discusses how FDA’s Orphan Drugs program helps in the fight against rare diseases. View the video below.

I run a brain tumor foundation.
We saw amazing results presented at the ASCO conference recently for the treatment of Glioblastoma multioforme (a rare disease – about 20,000 a year).
What makes these announcements so interesting is the lack of side effects/ toxicity, and some pretty amazing survivals.
For example, the ICT-107 vaccine trial (19 GBM patients) reported no grade 3 or 4 toxicity, and 2 year survival of 80%. PFS was 17.7 months compared to historical control of 6.9 months. This is with 3 simple injections with no side effects. Yet we are being told it is many years before patients can get access.
Another example: results of a large study of the Novo TTF device – in 237 patients randomized to the device alone or best of standard care, showed a slight edge in survival, but drastically lower side effects. It was approved in Europe, but USA patients can not get access..
How do we help speed these things up?

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